2

Safe harbor

This presentation and our remarks based upon it, including responses to questions made during and following the presentation, may include forward-looking statements. Such statements are subject to the risks and uncertainties we discuss in detail in our reports filed with the Securities & Exchange Commission, including in our quarterly report on Form 10-Q filed November 8, 2012. We expressly disclaim any obligation to release publicly any updates to forward-looking statements made during the course of this presentation.

3

Two late stage clinical programs

• Enobosarm (Ostarine; GTx-024), a SARM, for the prevention and treatment of muscle wasting in patients with non-small cell lung cancer: Eight clinical trials completed to date involving approximately 600

subjects

Input from with FDA, MHRA and MPA on our Phase III clinical development plan

Currently enrolling two pivotal Phase III clinical trials in non-small cell lung cancer (NSCLC) patients - topline results expected 1H 2013

DSMB reviewed safety data in May 2012 and October 2012 and agreed trial could continue as planned

• Capesaris (GTx-758), an oral selective ER alpha agonist, for secondary hormonal treatment of advanced prostate cancer: Eight clinical trials conducted to date involving approximately 500

subjects

FDA removed its Clinical Hold on Capesaris IND in May 2012

Phase II 712 clinical trial evaluating Capesaris® (GTx-758) for secondary hormonal treatment in men with metastatic castration resistant prostate cancer started 3Q2012

Selective Androgen Receptor Modulator (SARM)for the prevention and treatment of muscle wasting

in patients with lung cancer

5

SARM 2005

Enobosarm Approximately 600 patients have participated in eight clinical trials

GTx Enobosarm

GTx Enobosarm

GTx Enobosarm

GTx Enobosarm

GTx Enobosarm

Collaboration Enobosarm and Merck SARM

Collaboration Enobosarm

GTx Enobosarm

Phase I SAD

Phase I MAD

Phase II POC chronic sarcopenia

Phase IIb muscle wasting in cancer

Phase I divided dose

Phase Ib head to head chronic sarcopenia

Phase Ib PK study – Japanese women

Phase I Formulation study

2006 2007 2008 2009 2010 2011

Enobosarm increased lean body mass and improved physical function in three efficacy clinical trials

Phase IIb cancer cachexia trial:

159 subjects with cancer cachexia, 4 months tx

Phase II POC clinical trial:120 elderly men and postmenopausal women,3 months tx

Phase Ib sarcopenia trial:

88 postmenopausal women, 3 months tx

Morton, et al. AACR. 2009; abstract nr(9273). Steiner, et al. J Clin Oncol. 2010;28:7s(suppl;abstr 9147). Dalton, et al. J Cachexia Sarcopenia Muscle. 2011;2:153-161. Marcantonio, et al. Endocrine Reviews. 2010;31(3):(suppl 1)S872. 6

Chang

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rom

base

line

(kg

)C

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base

line

(kg

)

Chang

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base

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(kg

)

Chang

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rom

b

ase

line (

%)

Chang

e f

rom

base

line

(%)

Chang

e f

rom

base

line

(lb

s)

LEAN BODY MASS PHYSICAL FUNCTION

PlaceboEnobosarm 3 mg

1.6

1.4

1.2

1

0.8

0.6

0.4

0.2

0

-0.2

1.4

1.2

1

0.8

0.6

0.4

0.2

0

-0.2

25%

20%

15%

10%

5%

0%

-5%

-10%

-15%

30%

25%

20%

15%

10%

5%

0%

-5%

-10%

1.4

1.6

1.2

0.81

0.6

0.4

0.2

0

-0.2

-0.4

40

30

20

10

0

-10

-20

STAIR CLIMB

PlaceboEnobosarm 3 mg

STAIR CLIMB

PlaceboEnobosarm 3 mg

PlaceboEnobosarm 3 mg

BILATERAL LEG PRESS

PlaceboEnobosarm 3 mg

PlaceboEnobosarm 3 mg

PlaceboEnobosarm 3 mg

7

• At diagnosis, nearly 50% of advanced NSCLC patients have severe muscle loss and approximately 70% of NSCLC patients will lose muscle 88% have lower body functional limitations including the ability to

climb stairs, lift and carry 10 lbs, walk ¼ mile, and stoop, crouch or kneel

• Performance status is a predictor of a patient’s ability to tolerate chemotherapy, and poor performance status is a primary reason patients are not offered treatment

Performance status also predicts the likelihood of hospitalization, ability to maintain independence, and survival

Muscle wasting is an important cancer related symptom in patients with advanced NSCLC

Phase IIb clinical trial in cancer patients:Enobosarm increased lean body mass & improved physical function in NSCLC Subset analysis: 61 NSCLC patients; mean % weight loss at entry was 9.7%

Reported incidence of tumor progression similar across groups. The most common AEs were fatigue, anemia, nausea and diarrhea.

Steiner, et al. J Clin Oncol. 2011;29:15s(suppl;abstr 9022). 8

Lean Body MassLean Body Mass Physical FunctionPhysical Function

-20

-15

-10

-5

0

5

10

15

20

25

% c

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ine

placebo 1 mg 3 mg-1.5

-1

-0.5

0

0.5

1

1.5

Cha

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bas

elin

e (k

g)

P=0.198

-0.84

1.1

N=31

Placebo

Enobosarm17.6

N=31-14.7

Placebo

Enobosarm

P=0.051

Goal met in clinical trials

Ability to meet goal to be determined

• Advanced NSCLC is incurable

• With currently available platinum doublet therapies, median survival is 8–11 months and the one year survival rate is 30-40% for patients who maintain good physical function

Ramalingam et al, Ca Cancer J Clin, 2011; 61: 91-1129

Goals for the treatment of advanced stage NSCLC with chemotherapy

International pivotal Phase III clinical trials: POWER 1 and 2

Input from with FDA, MHRA (U.K.) and MPA (Sweden) on Phase III

clinical development plans

150 patients

150 patients

150 patients

Other endpoints•QoL – FAACT, FACIT fatigue scales•Healthcare resource

utilization•Adherence to chemo plans•Tolerance to chemo

Co-primary endpoints•Lean body mass•Physical function @ 3 months

Placebo

Enobosarm3 mg

Enobosarm 3 mg

Secondary endpoints•Durability of effect @ 5 months•Overall survival

Placebo

platinum + taxane

150 patients

platinum + non taxane

Indication: Prevention and treatment of muscle loss in patients with NSCLC• Stage III/IV NSCLC patients initiating 1st line chemotherapy

• Co-primary endpoints (responders analysis): (1) no loss of LBM; (2) at least 10% improvement in SCP

• Each endpoint α=0.05, power >93%

• Assumes 30% drop out rate by 3 months

10

EnobosarmAnticipated clinical development plan

Phase III-POWER 1Enobosarm 3 mg vs placebo in 300 pts

with NSCLC receiving platinum + taxane chemotherapy (5 month study)

Phase III-POWER 2Enobosarm 3 mg vs placebo in 300 pts

with NSCLC receiving platinum + non taxane chemotherapy (5 month study)

NDA

11

EnobosarmLarge market opportunity

• Lung cancer is the most common malignancy

Worldwide - estimated 1.6 million new cases and 1.4 million deaths each year

United States - 222,500 new cases and 157,300 deaths in 2010

50% present with advanced disease

• Indication being pursued is prevention and treatmentof muscle wasting in patients with NSCLC

• In US, 170,000+ advanced NSCLC patients initiate chemotherapy each year

• Currently, several drugs treating cancer related symptomsare priced between $30 and $50 per day

• GTx estimates muscle wasting in patients with NSCLCcould be a $750 million opportunity in US alone 12

EnobosarmLarge market opportunity

United States

Source: Datamonitor 2011; projected market size in 2012, US and EU Qualitative market research

Stage III-IV NSCLC is the majority of NSCLC diagnosed and, based on perceptions of oncologists, at least 50% of these patients have muscle wasting

hyperK+ Rates

NSCLC= 207 K

Stage III-IVNSCLC

= 161 K (78% of NSCLC)

Muscle Wasting

~50% or higher(81 K)

5 EU

hyperK+ Rates

NSCLC= 186 K

Stage III-IVNSCLC

= 148 K (79% of NSCLC)

Muscle Wasting

~50% or higher(74 K)

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GTx-024 Commercial Assessment & Forecast – May 2012

Oncologists believe that muscle wasting has a high to medium impact on a NSCLC patient’s physical activity, QoL, weight loss, ability to tolerate chemotherapy and

ultimately on survival

Source: US and EU Qualitative market research

EnobosarmLarge market opportunity

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GTx-024 Commercial Assessment & Forecast – May 2012

• Only 30% of US oncologists and 20% of European oncologists had any awareness of products in development for muscle wasting

• Oncologists in both the US and the EU expressed a high level of interest for new treatments for the management of muscle wasting Not interested

at all

Extremely interested

US(N=16)

5 EU(N=24)

Source: US Oncologist Quantitative and US and 5 EU Qualitative market research

There is a high level of interest in treatments for muscle wasting, but very little awareness of specific drugs in clinical development

US Quant

(N=150)

No needat all

HighNeed

EnobosarmLarge market opportunity

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Extremely interested

EnobosarmIntellectual property

• 79 enobosarm composition of matter and method of use patent applications approved or pending in U.S. and rest of world with expiration dates in 2024

• As a new chemical entity, issued patents should be eligible for patent term extension of up to 5 years (2029)

• GTx has 350 patents approved or pending worldwide for all SARMs including enobosarm

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Selective ERα agonist for the treatment of advanced prostate cancer

Evolving treatment paradigm Advanced prostate cancer

Hormonesensitive

FirstSecondaryHormone Tx

CapesarisEnzalutamide

Second Secondary Hormone Tx

CapesarisEnzalutamide

ThirdSecondary Hormone Tx

Abiraterone + PrednisoneCapesaris

Chemotherapy

DocetaxelPostChemotherapy

EnzalutamideAbiraterone/PrednisoneCabazitaxel

Castration Resistant Prostate Cancer (CRPC)

Advanced Prostate Cancer

750,000patients

100,000patients/

year

15,000patients/

year

Market

Italicized- not approved

*Potential for future

development

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Multiple potential mechanisms of action for a selective ERα agonist to treat prostate cancer

19

The relative amount of free T and T bound to albumin or SHBG in prostate cancer patients

Damber, JE et al, J. Endocrin. Invest, 6: 91-3, 1983

100

50

0

Tota

l te

stost

ero

ne %

Control Orchiectomy Estrogen

Free testosterone

SHBG bound testosterone

Albumin bound testosterone

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Bioavailable

Phase II studies in men with advanced prostate cancer confirms the mechanism of action• Phase II open label loading dose finding 705 clinical study in

advanced prostate cancer comparing1500mg BID and 1000mg BID loading doses followed by 1000mg or 2000mg maintenance doses (n=55)

• Castration rate greater than 90% for both arms• Capesaris increased SHBG and decreased free T

• Phase II open label maintenance dose finding 710 clinical study in advanced prostate cancer comparing Lupron, Capesaris 1000mg PO qD and Capesaris 2000mg PO qD (n=164) 2000mg Capesaris and Lupron arms

• Maintained castration by Kaplan-Meier estimates >95.5%• Similar testosterone escapes• Capesaris increased SHBG and decreased free T• Improvement in hot flashes, bone turnover markers and insulin

resistance

• Safety- VTE incidence rate increased for Capesaris21

Day 90

25 patients

Inclusion/Exclusion Criteria:• Serum PSA > 2ng/ml• Castrate (total T <50ng/dl)• ECOG 0-2• Maintain primary ADT

Capesaris 2000 mg PO q d

Endpoints:•Serum PSA response > 50% (Primary Endpoint)•Serum PSA Progression (PSA > 2 & >25%) •Serum free T and SHBG•Bone and Soft Tissue metastases

Phase II, secondary hormonal therapy 707 clinical study in chemotherapy naive CRPC

Serum PSA Response (N=7)

Serum PSA responders (>50% reduction)Mean SHBG =399% ± 85%

-90

-80

-70

-60

-50

-40

-30

-20

-10

0Day 1 Day 15 Day 30 Day 60

1S001

1S003

2S001

5S001

5S003

5S004

5S002Perc

en

t of

base

line

Days22

Phase II, open label, 712 clinical study of secondary hormonal treatment in men with metastatic CRPCstarted 3Q 2012

Subjects•mCRPC•Maintain ADT

Primary Endpoint:•Serum PSA response

Secondary Endpoints:•PSA progression•Progression free survival•Free T/SHBG•Adrenal (DHEA&DHEAS)•Estrogen deficiency side effects•SRE

23

Day 90

25 patients

25 patients

25 patients

GTx-758 250 mg

GTx-758 500 mg

GTx-758 125 mg

Day 0

30 d

30 d

23

Efficacy should be maintained with lower doses of Capesaris

Projected SHBG increases by dose and time*

*Based on Phase II 703/705 studies(Data on file)

Dose

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Capesaris Steering Committee

• Medical oncology Evan Yu* (U Washington) Johann DeBono* (Royal Marsden) Dan Petrylak* (Columbia/ Yale) Chuck Ryan* (UCSF) Phil Kantoff* (Dana Farber Harvard) Thomas Flaig* (U Colorado)

• Urology Badri Konety (U Minnesota)

• Advocacy group Tom Kirk (Us TOO)

* Confirmed participation

Phase IIb705 clinical study

Maintenance dose finding

Phase II710 clinical study

Loading dose finding

CapesarisAnticipated clinical development plan

Phase II712 clinical study

Secondary hormonal therapy in mCRPC patients (lower doses)

Phase II707 clinical study

Secondary hormonal therapy in CRPC patients

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CapesarisIntellectual Property

• 20 composition of matter and method of use patent applications and patents, which are either issued, allowed or pending in the US and rest of world with expiration dates of January 2029 in the US and November 2026 in the ROW

• As a chemical entity, issued US patent should be eligible for additional patent term extension of up to 5 years (for a maximum term of November 2034), as may be determined following FDA approval of Capesaris

Financial Summary

• Shares outstanding: 62.8 M

• Cash, cash equivalents and short-term investments at September 30, 2012: $47.3M

• Cash and short-term investments increased in October 2012 from the receipt of $19M in net cash proceeds from the sale of the rights to Fareston

• No debt, no warrants

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